Clinical Trial News - 10Mar2025

FDA News Release – FDA Roundup: March 11, 2025

11 March 2025

The FDA shared updates in its March 11, 2025, roundup. Key points include:

Data Integrity Concerns: A General Correspondence Letter was issued to Mid-Link Technology Testing Co., Ltd., which has submitted falsified or copied study data. The FDA has rejected all studies from this facility until it resolves these issues, urging manufacturers to independently verify third-party testing results.

Fat-Dissolving Injections Warning: The FDA issued six warning letters to companies selling unapproved fat-dissolving injectable drug products, emphasizing the potential harm of such products.

Biosimilar Approval: The FDA approved Omlyclo (omalizumab-igec) as the first interchangeable biosimilar to Xolair for treating allergic, inflammatory, and autoimmune conditions. It works by blocking IgE, the antibody responsible for allergic reactions.

Mutated DNA Restored to Normal in Gene Therapy Advance

Researchers successfully restored a mutated gene to its normal state for the first time, marking a breakthrough in gene therapy. In a small study by Beam Therapeutics, nine patients with alpha-1 antitrypsin deficiency (AATD)—a genetic disorder causing liver and lung damage—received a single infusion of lipid nanoparticles carrying a CRISPR-based gene editor. This editor precisely corrected the mutation by replacing a single incorrect DNA base.

Patients receiving the highest dose produced sufficient levels of the functional protein to potentially halt further organ damage. This innovative technique represents a significant step toward developing precise, one-time treatments for genetic diseases. Long-term follow-up will confirm its safety and lasting benefits.

Base editing, which corrects misspellings in DNA, offers an alternative to existing forms of gene therapy, which either add or remove things from the genome.

Australian man survives 100 days with artificial heart in world-first success

10 March 2025

An Australian man with severe heart failure has made history as the first person to leave a hospital with a total artificial heart implant. The BiVACOR total artificial heart, designed by Dr. Daniel Timms, uses magnetic levitation technology to replicate the natural blood flow of a healthy heart. This groundbreaking device served as a bridge for the patient, allowing him to live over 100 days with the implant before successfully receiving a donor heart transplant in early March.

The achievement has been hailed as a major medical milestone, with researchers emphasizing its potential to transform heart failure treatment globally. The BiVACOR heart aims to provide a life-saving alternative for patients awaiting transplants and, in the future, could even eliminate the need for donor hearts altogether.

FDA News Release – FDA Roundup: March 7, 2025

07 March 2025

The FDA's March 7, 2025, roundup highlighted two significant updates:

Approval of Encelto (revakinagene taroretcel-lwey), an allogeneic cell-based gene therapy approved for treating idiopathic macular telangiectasia type 2 (MacTel), a rare progressive eye disease. Administered through a single surgical procedure, it works by expressing recombinant human ciliary neurotrophic factor to support the survival of macular photoreceptors.

Expanded Use of Neffy (epinephrine nasal spray): Now approved for children aged 4 and older, weighing 15-30 kg, for emergency treatment of anaphylaxis. Originally approved in 2024 for individuals weighing 30 kg or more. This is the first nasal spray approved for anaphylaxis, offering a non-injection alternative.

J&J to stop studies of depression drug due to low effectiveness

06 March 2025

Johnson & Johnson has decided to halt late-stage trials of aticaprant, an experimental drug for major depressive disorder, due to insufficient effectiveness. This drug targeted anhedonia—a condition that diminishes the ability to feel pleasure. Neumora Therapeutics' navacaprant, a similar drug, also recently failed to show effectiveness in trials.

J&J will explore other uses for aticaprant in areas of unmet need, while continuing to develop its neuroscience portfolio, which includes the approved nasal spray Spravato and other treatments in development. Their recent $14.6 billion acquisition of Intra-Cellular Therapies underscores their commitment to brain disease treatments.

These universities have the most retracted scientific articles

27 February 2025

A first-of-its-kind analysis by Nature reveals which institutions are retraction hotspots.

Clinical Trials Market Size is Estimated to be US$ 149.58 Billion by 2034

19 February 2025

The global clinical trials market is projected to grow significantly, reaching an estimated size of $149.58 billion by 2034, up from $83.75 billion in 2024. This growth, at a compound annual growth rate (CAGR) of 6.10% from 2025 to 2034, is driven by increased investments in pharmaceutical R&D, a surge in drug candidates, and advancements in medical technology.

Key highlights include:

• North America held the largest market share (59.3%) in 2024, while Asia Pacific is expected to grow at the fastest rate (CAGR of 7.16%).
• The oncology segment contributed the largest market share (38.4%) in 2024.
• Interventional studies dominated with a 77.96% market share in 2024.
• Phase III trials accounted for the largest share (49.1%) in 2024.
• The market's expansion reflects the growing demand for innovative treatments and the globalization of clinical trials to include diverse patient populations.

New weight loss drugs aim to save muscle

19 February 2025

About a dozen drugmakers are developing new weight-loss treatments aimed at preserving muscle, and industry analysts, clinical trial experts and doctors say they may also need to demonstrate additional health benefits beyond the numbers on a scale to get approved.

Manual vs AI-Assisted Prescreening for Trial Eligibility Using Large Language Models—A Randomized Clinical Trial

17 February 2025

Patient identification and screening are persistent challenges in clinical trial recruitment and often result in delayed and incomplete patient enrollment, exacerbating the high cost of cardiovascular outcomes trials, which can be in hundreds of millions of dollars. Traditional manual screening methods are labor-intensive and may contribute up to one-third of clinical trial costs. Structured electronic health record queries improve the efficiency of clinical trial screening but still require manual review of unstructured data. Large language models (LLMs) parse unstructured data, which enables a shift in how patients are screened and enrolled. We developed an LLM tool, the Retrieval Augmented Generation Enabled Clinical Trial Infrastructure for Inclusion Exclusion Review (RECTIFIER), and demonstrated retrospectively that it can assess patient eligibility for specific inclusion and exclusion criteria with sensitivity, specificity, and accuracy exceeding that of study staff.

Genome Engineering Market To Reach USD 30.9 Billion by 2032, Growing at CAGR 16.7% | Agilent Technologies, Bluebird Bio, Boehringer Ingelheim

12 February 2025

The shift towards personal medicine is changing healthcare, running the demand for genome engineering. By taking advantage of advanced equipment such as CRISPR, scientists can now edit genes with remarkable accuracy to develop cancer, rare diseases and adapted treatment for chronic conditions. The promise of more effective, patient-specific remedies has made this approach highly attractive to researchers, healthcare providers and pharmaceutical companies. This increased attention on accurate therapy not only improves patient results, but also improves innovation, promotes growth in the genome engineering market as the medical industry strives to deal with diseases on its genetic roots.

Genome engineering increases moral concerns, especially when it involves human germinal editing. The possibility of creating "designer infants" or inadvertently transforms future generations increases the widespread debate. In addition, strict rules limit certain research areas and slow down the speed of innovation. Public apprehensions around safety requirements and security and current obstacles for both researchers and companies’ misuse. These moral and legal complications prevent the entire capacity of the market, forcing stakeholders to navigate a highly regulated environment to balance innovation with moral ideas and gain public belief.